Stock drops after development setback
Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) shares fell 14% on Tuesday after the company announced it will discontinue development of tebapivat for sickle cell disease following results from a Phase 2 clinical trial.
The biotechnology company concluded that the study did not produce sufficiently differentiated results to justify advancing the programme.
Phase 2 study falls short of development criteria
The Phase 2 trial enrolled 59 patients aged 16 years and older with sickle cell disease and evaluated tebapivat, an oral pyruvate kinase activator, over a 12-week treatment period.
The primary endpoint assessed hemoglobin response, defined as an increase of at least 1.0 g/dL in average hemoglobin levels during Weeks 10 through 12 compared with baseline.
Response rates reached 43.8% in the 2.5 mg once-daily group, 47.1% in the 5.0 mg group and 29.4% in the 7.5 mg group, compared with 33.3% for patients receiving placebo.
Although the study showed improvements in hemoglobin levels and markers of hemolysis across all tebapivat dose groups, Agios said the findings did not demonstrate enough differentiation to support continued investment in the programme.
Safety profile remains consistent
The company stated that the safety and tolerability profile of tebapivat was consistent with previous clinical studies conducted in patients with sickle cell disease.
Focus shifts to mitapivat
Agios will now continue to prioritise mitapivat, its other pyruvate kinase activator, which remains under FDA Priority Review for the treatment of sickle cell disease.
Earlier this month, the U.S. Food and Drug Administration accepted the company’s supplemental New Drug Application seeking accelerated approval for mitapivat. The application has a Prescription Drug User Fee Act (PDUFA) target action date of November 1, 2026.
Agios Pharmaceuticals stock price
